Gene therapy refers to the technology of modifying or manipulating gene expression or changing the biological characteristics of living cells to achieve disease treatment. According to the timing of gene change, approaches of gene therapy can be divided into two categories: ex vivo and in vivo.
Ex vivo is to repair the genes of cells in culture, and then import the repaired cells into the human body; while in vivo is to directly supply the materials that can change the genes into the human body through the use of a vector.
Animal models constructed with gene-editing technology are a commonly used in vivo approach, these may be used not only to simulate the physiological and pathological environment of human body, but also to simulate the pathological changes of some diseases, due to its potential for high consistency with the development process of diseases.
At present, gene therapy products are used to treat a variety of diseases, including cancer, genetic disorders, and infectious diseases. However, the risks of gene therapy, such as severe immune response (like cytokine storm), neurotoxicity, and insertion mutation (tumorigenicity) have influenced the effectiveness and safety of gene therapy treatments. To minimize the occurrence of these risks, a lot of preclinical research work is needed to lay the foundation for the treatment before applied to human beings. This includes the in vivo animal model construction, vector selection, treatment strategy determination, and more.